WO2015030616A3 - Use of nucleic acid molecules in therapy of diseases induced by expansion of trinucleotide cag repeats - Google Patents

Use of nucleic acid molecules in therapy of diseases induced by expansion of trinucleotide cag repeats Download PDF

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Publication number
WO2015030616A3
WO2015030616A3 PCT/PL2014/000100 PL2014000100W WO2015030616A3 WO 2015030616 A3 WO2015030616 A3 WO 2015030616A3 PL 2014000100 W PL2014000100 W PL 2014000100W WO 2015030616 A3 WO2015030616 A3 WO 2015030616A3
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WO
WIPO (PCT)
Prior art keywords
expansion
nucleic acid
therapy
acid molecules
diseases induced
Prior art date
Application number
PCT/PL2014/000100
Other languages
French (fr)
Other versions
WO2015030616A2 (en
WO2015030616A4 (en
Inventor
Wlodzimierz Krzyzosiak
Marta OLEJNICZAK
Paulina GALKA-MARCINIAK
Agnieszka Fiszer
Original Assignee
Instytut Chemii Bioorganicznej Pan
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Instytut Chemii Bioorganicznej Pan filed Critical Instytut Chemii Bioorganicznej Pan
Priority to US14/916,039 priority Critical patent/US9970004B2/en
Priority to EP14781962.7A priority patent/EP3041936A2/en
Publication of WO2015030616A2 publication Critical patent/WO2015030616A2/en
Publication of WO2015030616A3 publication Critical patent/WO2015030616A3/en
Publication of WO2015030616A4 publication Critical patent/WO2015030616A4/en
Priority to US15/954,561 priority patent/US10329566B2/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Subjects of the invention are: nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG-type repeats. Solution relates to the new concept of treating hereditary human neurological diseases caused by expansion of CAG-type trinucleotide repeats using RNA interference technology.
PCT/PL2014/000100 2013-09-02 2014-09-02 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of rna interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats WO2015030616A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
US14/916,039 US9970004B2 (en) 2013-09-02 2014-09-02 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interferencde in eukaryotic host and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG repeats
EP14781962.7A EP3041936A2 (en) 2013-09-02 2014-09-02 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of rna interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats
US15/954,561 US10329566B2 (en) 2013-09-02 2018-04-16 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interference in eukaryotic host and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG repeats

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
PL405224A PL243776B1 (en) 2013-09-02 2013-09-02 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, transgenic mammal, method for inducing RNA interference in an eukaryotic host and application of nucleic acid molecule in the therapy of diseases induced by the expansion of trinucleotide repetitions of CAG type
PLP405224 2013-09-02

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US14/916,039 A-371-Of-International US9970004B2 (en) 2013-09-02 2014-09-02 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interferencde in eukaryotic host and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG repeats
US15/954,561 Division US10329566B2 (en) 2013-09-02 2018-04-16 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interference in eukaryotic host and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG repeats

Publications (3)

Publication Number Publication Date
WO2015030616A2 WO2015030616A2 (en) 2015-03-05
WO2015030616A3 true WO2015030616A3 (en) 2015-09-03
WO2015030616A4 WO2015030616A4 (en) 2015-10-01

Family

ID=51688382

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/PL2014/000100 WO2015030616A2 (en) 2013-09-02 2014-09-02 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of rna interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats

Country Status (4)

Country Link
US (2) US9970004B2 (en)
EP (1) EP3041936A2 (en)
PL (1) PL243776B1 (en)
WO (1) WO2015030616A2 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017190197A1 (en) * 2016-05-05 2017-11-09 Benitec Biopharma Limited Reagents for treatment of hepatitis b virus (hbv) infection and use thereof
AU2019385598A1 (en) * 2018-11-19 2021-06-03 Uniqure Ip B.V. RNAi induced reduction of ataxin-3 for the treatment of Spinocerebellar ataxia type 3
IL311638A (en) * 2021-10-06 2024-05-01 Board Of Trustees Of Southern Illinois Univ Compositions and methods for treating cag repeat diseases

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003013437A2 (en) * 2001-08-07 2003-02-20 University Of Delaware Compositions and methods for the prevention and treatment of huntington's disease
WO2006031267A2 (en) * 2004-06-02 2006-03-23 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
WO2011097388A1 (en) * 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression
WO2012109667A1 (en) * 2011-02-12 2012-08-16 University Of Iowa Research Foundation Therapeutic compounds
WO2013033223A1 (en) * 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003091433A1 (en) * 2002-04-26 2003-11-06 National Institute Of Advanced Industrial Science And Technology EXPRESSION SYSTEMS FOR STEM LOOP RNA MOLECULE HAVING RNAi EFFECT
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003013437A2 (en) * 2001-08-07 2003-02-20 University Of Delaware Compositions and methods for the prevention and treatment of huntington's disease
WO2006031267A2 (en) * 2004-06-02 2006-03-23 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
WO2011097388A1 (en) * 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression
WO2012109667A1 (en) * 2011-02-12 2012-08-16 University Of Iowa Research Foundation Therapeutic compounds
WO2013033223A1 (en) * 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
A. FISZER ET AL: "Self-duplexing CUG repeats selectively inhibit mutant huntingtin expression", NUCLEIC ACIDS RESEARCH, vol. 41, no. 22, 1 December 2013 (2013-12-01), pages 10426 - 10437, XP055166819, ISSN: 0305-1048, DOI: 10.1093/nar/gkt825 *
AGNIESZKA FISZER ET AL: "An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases", BMC MOLECULAR BIOLOGY, vol. 13, no. 1, 7 March 2012 (2012-03-07), pages 6, XP021124662, ISSN: 1471-2199, DOI: 10.1186/1471-2199-13-6 *
DONGBO YU ET AL: "Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression", CELL, vol. 150, no. 5, 1 August 2012 (2012-08-01), pages 895 - 908, XP055166558, ISSN: 0092-8674, DOI: 10.1016/j.cell.2012.08.002 *
HU J ET AL: "Allele-Selective Inhibition of Huntingtin Expression by Switching to an miRNA-like RNAi Mechanism", CHEMISTRY AND BIOLOGY, vol. 17, no. 11, 24 November 2010 (2010-11-24), pages 1183 - 1188, XP027511220, ISSN: 1074-5521, DOI: 10.1016/J.CHEMBIOL.2010.10.013 *

Also Published As

Publication number Publication date
US10329566B2 (en) 2019-06-25
US20160376586A1 (en) 2016-12-29
PL243776B1 (en) 2023-10-09
EP3041936A2 (en) 2016-07-13
US20190024081A1 (en) 2019-01-24
WO2015030616A2 (en) 2015-03-05
PL405224A1 (en) 2015-03-16
US9970004B2 (en) 2018-05-15
WO2015030616A4 (en) 2015-10-01

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